Abstract Introduction Narcolepsy type 1 (NT1) and type 2 (NT2) are chronic neurological disorders characterized by excessive daytime sleepiness, disrupted nocturnal sleep, and symptoms of dissociated REM. Sodium oxybate has been a mainstay of treatment, beginning with FDA approval of twice-nightly oxybate (TN-OXB) in 2002. Once-nightly oxybate (ON-OXB) received FDA approval in 2023 to eliminate middle-of-the-night dosing. We sought to evaluate patient adherence and tolerance with ON-OXB in the clinical setting. Methods A manual chart review was performed for patients seen at a sleep center in Sugar Land, TX from 6/1/2023 to 12/7/2025. Eligible patients carried a diagnosis of NT1/NT2 and had been prescribed ON-OXB. EMR reporting tools were used to identify charts with a ON-OXB prescription, followed by manual confirmation of inclusion criteria and gathering of demographic information. Patients who had not returned for follow-up after receiving an ON-OXB prescription were excluded. Results Seventy-six patients were initiated on ON-OXB: 31 NT1 (41%), 45 NT2 (59%); 58 female, 18 male. Of these, 37 (49%) were oxybate naive, and 39 (51%) transitioned from TN-OXB. Twenty seven (35.5%) patients stopped ON-OXB. Of the oxybate naive, 26 (70%) remained on therapy. Of those who switched from TN-OXB, 23 (59%) remained on ON-OXB. A higher percentage of NT1 patients remained on ON-OXB than NT2 patients (74% vs. 58%, respectively). Women found more success with ON-OXB than men (67% vs. 56%, respectively). Of the 27 who stopped, 15 (55.5%) went to TN-OXB (6 oxybate naive, 9 switched back). Twelve (15.8%) patients outright discontinued oxybate. Conclusion In this real-world clinical setting, continuation of ON-OXB therapy was lower than reported in clinical trials. Differences could be seen in narcolepsy type, prior oxybate exposure, and gender. While most patients did remain on ON-OXB (64.5%), 19.7% went to TN-OXB, and 15.8% discontinued oxybate. These trends show that while an increase in treatment alternatives is beneficial, additional options are needed. This study is limited by small sample size. Larger studies are needed to confirm these trends. Support (if any) N/A
Colwell et al. (Fri,) studied this question.
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